Asciminib and Inotuzumab Ozogamicin for Relapsed or Refractory Philadelphia Chromosome-positive B-cell Acute Lymphoblastic Leukemia (R/R Ph+ B-ALL)

November 9, 2026 updated by: Sunil G. Iyer

A Phase I Open-label, Multicenter Trial of Asciminib (ABL001) Added to Inotuzumab Ozogamicin (Besponsa®) in Patients With Relapsed or Refractory Philadelphia Chromosome-positive B-cell Acute Lymphoblastic Leukemia

This is a multi-center phase 1 open-label study to determine the safety of ABL001 and INO in patients with relapsed or refractory Philadelphia chromosome-positive b-cell acute lymphoblastic leukemia.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This study will test a combination of two drugs for people with Philadelphia chromosome-positive B-cell acute lymphoblastic leukemia (Ph-positive B-ALL), a fast-growing blood cancer. Asciminib is a targeted oral drug that is FDA-approved for chronic myeloid leukemia, but is not FDA-approved for Ph-positive B-ALL. Inotuzumab ozogamicin (also called Besponsa) is an FDA-approved treatment that uses an antibody (a protein) to deliver a small amount of chemotherapy directly to leukemia cells. It is commonly used when leukemia has come back after earlier treatment (relapsed) or has not improved with treatment (refractory). This study aims to find the safest dose of asciminib when it is given together with inotuzumab ozogamicin. The goal is to see whether this combination can provide a more effective and better tolerated treatment option for people whose leukemia has returned or has not responded to previous therapies.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
45

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • United States
    • Florida
      • Miami, Florida, United States, 33136
        • Not yet recruiting
        • University of Miami Sylvester Comprehensive Cancer Center
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60805
        • Not yet recruiting
        • University of Chicago Comprehensive Cancer Center
        • Contact:
    • New York
      • New York, New York, United States, 10032
    • Washington
      • Seattle, Washington, United States, 98109
        • Not yet recruiting
        • University of Washington Fred Hutchinson Comprehensive Cancer Center
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Documented and confirmed CD22+ R/R Ph+ B-ALL according to the World Health Organization (WHO) classification

    • Relapsed ALL is defined as the detection of ≥ 5% blasts in the bone marrow with or without non-central nervous system (CNS) extramedullary disease
    • Refractory ALL is defined as the failure to obtain a CRc after at least two courses of intensive induction or immunotherapy such as blinatumomab, or to CAR-T cells. Persistent MRD without ≥ 5% blasts in the bone marrow will not be sufficient for inclusion

  2. Disease must be positive for Ph (p190 or p210 transcript) by cytogenetics or PCR

  3. Disease must be positive for CD22, defined as any percentage of leukemic blasts expressing CD22

  4. Disease must be positive for t(9;22) by Cytogenetics and FISH. Subjects with complex translocations resulting in BCR::ABL1 fusion protein are eligible.

  5. BCR-ABL mutational panel performed within 90 days prior to start of treatment, including the following 4 mutations: T315I and M244V, A337V, or F359C/I/V mutations. Specific panel may be determined per institution, however must include all aforementioned mutations. Note: T315I mutated patients are only eligible once Dose Level 3 is open to enrollment

  6. Subject must have recovered (no worse than Grade 1 severity) from effects of all previous treatments.

  7. Able to provide informed consent

  8. Age ≥18 years of age

  9. Eastern Cooperative Oncology Group (ECOG) performance score of 0 to 2 or Karnofsky ≥60%

  10. Adequate organ function defined by the following parameters:

    • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5x the upper limit of normal (ULN)
    • Total bilirubin ≤1.5x the ULN unless due to known Gilbert's disease or hemolysis due to blood transfusion
    • For patients with Gilbert's disease: total bilirubin ≤ 3x the ULN
    • Calculated glomerular filtration rate (GFR) of ≥ 15 mL/min/1.73 m2

  11. Female patients of childbearing potential must not be nursing or planning to become pregnant

  12. Female patients of childbearing potential require a negative urine or serum pregnancy test within 30 days of study therapy.

  13. Female patients of childbearing potential must be willing to use at least 1 method of highly effective contraception during the study until 8 months after last dose of inotuzumab ozogamicin, or 1 week after the last dose of asciminib, whichever is later.

  14. Male patients who are sexually active must be willing to use condoms and refrain from sperm donation during the study until 5 months after last dose of inotuzumab ozogamicin

  15. Willing to provide pretreatment bone marrow aspirate and biopsies samples as well as subsequent bone marrow aspirate and biopsies samples during the study.

Exclusion Criteria:

  1. Presence of severe and/or uncontrollable concurrent medical disease that in the opinion of the investigator could cause unacceptable safety risks or compromise compliance with the protocol (e.g. uncontrolled diabetes, active or uncontrolled infection)
  2. All prior Philadelphia chromosome-directed TKI therapy must be discontinued at least five days prior to study entry; corticosteroids and hydroxyurea are permitted during this period
  3. Treatment with chemotherapy, radiotherapy, or biologic therapy within 3 weeks prior to treatment Note: Hydroxyurea or corticosteroids is permitted; Previous treatment with ≥1 BCR-ABL-directed TKI
  4. Prior allogeneic stem cell transplant < 90 days prior to anticipated treatment start
  5. Patients with active GVHD (graft versus host disease) and/or receiving immunosuppressive drugs for GVHD
  6. Patients receiving calcineurin inhibitors < 4 weeks prior to anticipated treatment start
  7. Ph-directed TKI within 5 days of treatment start
  8. Prior exposure to INO
  9. Prior exposure to asciminib
  10. The presence of mixed phenotype acute leukemia or CML in lymphoid blast phase
  11. Presence of BCR-ABL1 M244V, A337V, or F359C/I/V mutations other n-lobe known to confer clinical resistance to asciminib as detected by ABL1 kinase mutational panel
  12. Patients with T315I mutation as detected by ABL1 kinase mutational panel are excluded from asciminib dose levels lower than 200mg BID
  13. Clinical suspicion for active CNS involvement by ALL; previously treated CNS involvement is allowed
  14. Second malignancy requiring treatment within 6 months of study enrollment, with the exception of non-melanoma skin cancers or cancers requiring hormonal therapy only
  15. Active, uncontrolled infection
  16. Human immunodeficiency virus (HIV) not controlled by standard therapy
  17. Active hepatitis B or C infection. Participants whose infections are controlled with antiviral therapy are allowed.
  18. Patients with end stage renal disease requiring dialysis

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Single-Arm

All participants enrolled to the treatment arm of the study will receive Asciminib in combination with Inotuzumab Ozogamicin.

INO is to be dosed in a fractionated schedule as follows:

  • Prior to achieving compositive complete remission (CRc): 0.8 mg/m2 day 1, 0.5 mg/m2 days 8 and 15
  • Once CRc achieved (even if MR3/MR4 not achieved): 0.5 mg/m2 days 1, 8, 15

Asciminib will be administered at a dose of 40, 60, 80, 160, or 200 mg twice daily.
Drug: Asciminib

Asciminib is a first-in-class STAMP (Specifically Targeting the ABL Myristoyl Pocket), and is an approved TKI in newly-diagnosed and previously-treated chronic myeloid leukemia (CML). It is a non-chemotherapeutic, oral agent which specifically and potently targets the mutant Philadelphia chromosome driven by t(9;22). Due to its novel mechanism of action as a STAMP, asciminib is active in the setting of mutations in the BCR-ABL ATP-binding pocket including the T315I gatekeeper mutations, which confers resistance to first- and second-generation tyrosine kinase inhibitors (TKIs). Asciminib has not yet been studied in Philadelphia-chromosome positive B-ALL.

Other Names:
  • ABL001
Drug: Inotuzumab ozogamicin

Inotuzumab ozogamicin (INO) is an antibody-drug conjugate which targets CD22, an antigen present in >90% of B-ALL cases. INO is FDA approved and a standard-of-care treatment in relapsed/refractory B-ALL. The drug is approved and active in Ph-positive and -negative disease.

Other Names:
  • INO
  • BESPONSA

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of adverse events
Time Frame: 36 Months

The safety profile of asciminib added to inotuzumab ozogamicin (INO) will be evaluated by the rates of adverse events categorized by the NCI Common Terminology Criteria for Adverse Events [CTCAE] v5.0.
36 Months
Maximum Tolerated Dose (MTD) of asciminib
Time Frame: 36 Months

The MTD of asciminib will be determined using a dose escalation strategy. The MTD is defined as the highest dose that be administered without causing dose-limiting toxicities in 75% of participants.
36 Months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Composite Complete Response (CRc) rate
Time Frame: 36 Months

Composite complete response (CRc) is defined as the rate of complete response (CR) + complete response with incomplete hematologic recovery (CRi), with complete metabolic response by PET/CT by the end of consolidation, according to the European LeukemiaNet (ELN) 2024 ALL criteria.
36 Months
Duration of remission (DOR)
Time Frame: 36 Months

DOR is defined as the time from CRc until relapse or death from any cause, whichever is first
36 Months
Overall survival (OS)
Time Frame: 36 Months

OS is defined as the time from study entry until death from any cause
36 Months
Event-free survival (EFS)
Time Frame: 36 Months

EFS is defined as the time from randomization to an event which may include disease progression, discontinuation of the treatment for any reason, or death
36 Months
Rate of molecular response (MR)
Time Frame: 36 Months

The rate of molecular response (MR) is assessed by clearance of measurable disease <0.1% (MR3) and <0.01% (MR4) as measured by BCR::ABL1 PCR in the bone marrow with complete metabolic response by PET/CT by the end of consolidation.
36 Months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Sunil G. Iyer

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Novartis

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Sunil Iyer, MD, Columbia University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 31, 2027

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 31, 2030

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2030

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 27, 2026

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 9, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 13, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 13, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 9, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ACYY0048

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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